A pill containing millions of bacteria ready to colonize your gut might be a nightmare to many. But it may become an effective new tool for fighting disease.
In many inherited genetic diseases a mutated gene means that an individual cannot make a vital substance necessary for their body to grow, develop, or function. Sometimes this can be fixed with a synthetic substitute — a pill — that they can take daily to replace what their body should have made naturally. People with a rare genetic disease called phenylketonuria (PKU) lack an enzyme that is essential for breaking down protein. Without it, toxic chemicals build up in the blood and can cause permanent brain damage.